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Journal Articles

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

David Salas, Karin L. Kwikkers, Nerea Zabaleta, Andrea Bazo, Harald Petry, Sander J. van Deventer, Gloria Gonzalez Aseguinolaza, Valerie Ferreira
Journal: Blood Advances
Blood Adv (2019) 3 (17): 2632–2641.
https://doi.org/10.1182/bloodadvances.2019000380
Published: 2019
Includes: Supplemental data
Journal Articles

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

Ornellie Bernadin, Fouzia Amirache, Anais Girard-Gagnepain, Ranjita Devi Moirangthem, Camille Lévy, Kuiying Ma, Caroline Costa, Didier Nègre, Christian Reimann, David Fenard, Agata Cieslak, Vahid Asnafi, Hanem Sadek, Rana Mhaidly, Marina Cavazzana, Chantal Lagresle-Peyrou, François-Loïc Cosset, Isabelle André, Els Verhoeyen
Journal: Blood Advances
Blood Adv (2019) 3 (3): 461–475.
https://doi.org/10.1182/bloodadvances.2018027508
Published: 2019
Includes: Supplemental data
Journal Articles

Protein kinase C signaling dysfunction in von Willebrand disease (p.V1316M) type 2B platelets

Caterina Casari, David S. Paul, Sophie Susen, Cécile Lavenu-Bombled, Annie Harroche, Raymond Piatt, Kathryn O. Poe, Robert H. Lee, Marijke Bryckaert, Olivier D. Christophe, Peter J. Lenting, Cécile V. Denis, Wolfgang Bergmeier
Journal: Blood Advances
Blood Adv (2018) 2 (12): 1417–1428.
https://doi.org/10.1182/bloodadvances.2017014290
Published: 2018
Includes: Supplemental data
Journal Articles

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency

Tayebeh Soheili, Amandine Durand, Fernando E. Sepulveda, Julie Rivière, Chantal Lagresle-Peyrou, Hanem Sadek, Geneviève de Saint Basile, Samia Martin, Fulvio Mavilio, Marina Cavazzana, Isabelle André-Schmutz
Journal: Blood Advances
Blood Adv (2017) 1 (27): 2781–2789.
https://doi.org/10.1182/bloodadvances.2017012088
Published: 2017
Journal Articles

Hemophilia gene therapy comes of age

Lindsey A. George
Journal: Blood Advances
Blood Adv (2017) 1 (26): 2591–2599.
https://doi.org/10.1182/bloodadvances.2017009878
Published: 2017
Journal Articles

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, Solenne Marmier, Christian Leborgne, Fanny Collaud, Marcelo Simon Sola, Severine Charles, Giuseppe Ronzitti, Alban Vignaud, Laetitia van Wittenberghe, Beatrice Marolleau, Fabienne Jouen, Sisareuth Tan, Olivier Boyer, Olivier Christophe, Alain R. Brisson, Casey A. Maguire, Federico Mingozzi
Journal: Blood Advances
Blood Adv (2017) 1 (23): 2019–2031.
https://doi.org/10.1182/bloodadvances.2017010181
Published: 2017
Includes: Supplemental data